From The Editor | August 8, 2017

DIA Trends: Technology, Trial Design, & Regulators

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical

DIA Trends: Technology, Trial Design, & Regulators

In his role as SVP and managing director, Americas for DIA Global, Sudip Parikh has the opportunity to look across the pharmaceutical industry at what companies are doing in regard to clinical trials, from those being performed at academic institutions up to the large, and more complicated, global trials being conducted at Big Pharma. He also has insights into what the industry can do to make trials more efficient and speed up the entire trial process.

“Many of the best practices I see pertaining to clinical trials seem to flow from three buckets,” says Parikh. “The first bucket relates to the methodologies and trial designs available to clinical trials. Today we are moving beyond the ‘traditional’ randomized clinical trial (RCT) and moving more towards adaptive trials and the use of real-world evidence. Some of these designs are modified on-the-fly. Others recruit patients who may participate in not just one trial, but multiple trials. We are also seeing greater use of master protocols that can test multiple drugs at one time. These and others are all trial designs that have not been used a great deal in the past.”  

The second bucket is new technologies, especially those pertaining to data. With data now populating databases from clinics, sensors, electronic health records, and other sources, there are efficiencies and - more importantly - insights that can be learned from the IT world. Parikh finally sees these efficiencies making their way into clinical trials. He believes academia, sponsor companies, and CROs are finding new and better ways to include real-world data in their clinical trials.

Parikh cites culture as the third bucket. He notes that regulatory agencies are now much more open to having discussions about trial designs and every element of a submission. Parikh does not believe we would be seeing the move towards real-world data were it not for regulators being receptive.

“We are now seeing real acknowledgement from the FDA that technology and methodology are advancing, and regulatory policy has to keep up. I tend to refer to these policy changes as innovations in their own right, because they involve doing things that have not been tried before. But, it is really a cultural change happening within both companies as well as regulators.”

Another area where we are seeing cultural change is the move towards clinical trials that are more patient-centric. Parikh is encouraged by the trend of both sponsors and FDA looking at trials from the perspective of patients. That includes taking into account how difficult it is to participate in a trial and looking at end points from the patient perspective.

Add It All Together

So, what does it mean if we combine all three of these trends? Parikh believes they are leading to a new set of best practices in the clinical space. Looking back over just the last 10 years, he has seen wonderful progress being made in improving clinical trials. However, much remains to be done.

The end result we all hope to see is approved products getting to patients faster. In that regard the results, at least thus far, have been spotty. Parikh sees new oncology products moving through pipelines rather quickly, while some rare disease products seem to be taking longer. Still, he is not discouraged. Even if a rare disease treatment is moving slowly, at least we are making positive progress.  

“As a whole, remarkable progress is being made in all parts of our ecosystem,” he says. “I would also add that ‘culture eats strategy for breakfast,’ to use a quote from Peter Drucker that still holds true today. Trial design and technology mean nothing if that is our only focus. As an industry, if we do not get that cultural element right, that handshake between industry, regulators, and patients, we will not make progress. And today, increasingly, we need to include payers as well. We need to keep making more progress on the cultural side to ensure that trial conduct matches current needs.”

Are You Staying On Top Of It?

It would be a challenge for the industry if any one of these three things (trial designs, technology, and culture) were changing. We are witnessing all three things changing quickly and at the same time. With all of this change taking place simultaneously, are notoriously conservative pharma companies keeping up?

“It’s a lot for some of them to keep up with,” says Parikh. “If you talk to the executives within large pharma companies about current trends, they will mention all of these topics. But if you dig down deeper into these organizations, you find the topics are not discussed as much. It’s not necessarily a disconnect; trial focus is simply not the same at different levels of the company.”

Budgets are always a concern, but the goals of the folks running clinical trials still have to match up with the goals of upper-level executives. That can be challenging in large organizations. There is a lag between the time that leaders see the need for change and that information making its way to the point where those changes can be implemented.

“You can see that lag when you talk to the folks who are running large global trials,” states Parikh. “The constraint of goals and budgets means it takes time for trials to change in order to meet new corporate guidance. Patient centricity is a good example. If you want to make sure you are incorporating the patient voice into your trials, there will be a cost associated with that. Unfortunately, it can also take time and may not immediately appear to align with another one of your primary outcomes, which is getting treatments to patients faster. Someone in charge of the trial will have to decide what outcomes are the most important. Eventually we will get to the point where we have both greater centricity and efficiency, but we have to understand it will take some time. We have made a lot of progress, and things will continue to get better.”

Finally, Parikh cautions companies to not invest heavily in new technologies at the expense of overly complicating trials. For guidance, he recommends always putting your focus on the patient-centric aspect of trials.

“Always ask yourself what is important and valuable to the patient,” he adds. “The answer to that question should be providing the framework to make technology decisions. Will it help get the treatment to the patient faster? Will it result in higher adherence? Will it make the treatment user-friendly? By answering those questions through the eyes of the patient, companies will make the right decisions.”